On November 20, 2018 LEO Pharma, a Danish company specializing in dermatology, and PellePharm, California’s forerunner of rare diseases, reported a strategic development and commercialization collaboration to address unmet medical needs various skin diseases without approved drugs and to promote innovation and access to potential therapies for patients with life-changing diseases such as Gorlin-Goltz syndrome and high-frequency basal cell carcinoma (BCC), two distinct and rare skin cancers (Press release, Leo, NOV 20, 2018, View Source [SID1234531516]).
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" We are very pleased to partner with PellePharm, the pioneers of Gorlin-Goltz Syndrome and experts in rare skin diseases. It supports our ambitious strategy for 2025 and marks the entry of LEO Pharma into the field of rare skin diseases. It also provides a unique opportunity to further develop the first compound to treat people with very severe skin conditions who are currently not receiving any therapies, "said Thorsten Thormann, Vice President of Research at LEO Pharma.
Under the terms of the agreement, LEO Pharma has initially committed $ 70 million of equity and research and development R & D to support the global Phase 3 study for the topical gel, patidegib, 2% To finance the prevention and treatment of Gorlin-Goltz syndrome, with LEO Pharma securing an option to acquire all of its shares in PellePharm. In addition, PellePharm and its shareholders could receive up to $ 690 million including a merger payment and milestone payments for approval and commercialization. In addition, after achieving certain commercial milestones, PellePharm shareholders are entitled to a double-digit royalty payment.
" Our company is committed to fighting rare dermatological diseases at the source and providing patients with new breakthrough treatments as efficiently and effectively as possible. As the world leader in the field of medical dermatology, LEO Pharma is the ideal development and marketing partner. This collaboration paves the way for our pivotal Phase 3 Gorlin-Goltz syndrome study in early 2019. Following the potential merger, we look forward to working with LEO Pharma on other rare skin diseases with unmet needs, "said Sanuj Ravindran, President and Chief Executive Officer of PellePharm.
Under the terms of the agreement, a Joint Development Committee will be set up, where PellePharm will be responsible for global development and LEO Pharma will have an advisory role. Both companies will jointly push ahead with marketing planning and LEON Pharma’s Chief Financial Officer Anders Kronborg will join PellePharm’s Board of Directors.
"The Gorlin Syndrome Group welcomes the news about the collaboration between PellePharm and LEO Pharma in developing patidegib with topical application for BCC treatment. Data from the PellePharm Phase 2 study suggest that patidegib is an effective BZK treatment with minimal side effects. We hope that by working with LEO these benefits will be confirmed in a Phase 3 study. An effective topical treatment for the prevention or treatment of BCC could prevent the painful, disruptive and disfiguring surgical procedures that we are currently undergoing, "said Sally Webster, chairperson, and Matthew Helbert, trustee of the Gorlin Syndrome Group UK
There are currently no FDA-approved therapies for Gorlin-Goltz syndrome and surgery is the treatment standard for this rare disease. Sometimes patients with this lifelong, serious illness have to undergo up to 30 surgeries per year from the middle age of teens.
PellePharm’s topical formulation (2%) is expected to be the first approved therapy for the prevention of Gorlin-Goltz syndrome, also known as nevoid cell basal cell carcinoma nevus syndrome (BCCNS), and has entered Phase 2 clinical trials has shown promise in treating patients with Gorlin-Goltz syndrome and sporadic BCC in the US and the UK.
" For more than 18 years, our organization has been dedicated to helping, educating, and finding much-needed preparations for people with Gorlin-Goltz Syndrome (BCCNS), and yet there are still no FDA-approved therapies for this serious disease," said Jean Pickford, executive director of the BCCNS Alliance. " Especially against the background of the Patidegib Phase 3 trial, which will begin recruitment in early 2019, PellePharm’s commitment – now in partnership with LEO Pharma – is very encouraging to our community."
PellePharm was advised by Rothschild & Co. on this transaction.
About Patidegib
The topical gel patidegib has been shown to be promising in a phase 2 clinical trial for the relief of BZK tumors in Gorlin-Goltz syndrome by blocking the hedgehog signaling pathway and thereby controlling the disease at the source. The topical formulation of patidegib is expected to achieve the efficacy already demonstrated for oral patidegib in Phase 1 studies without the occurrence of negative systemic side effects. The gel formulation is stable at room temperature for at least two years, so this could be a realistic treatment option for ongoing home-based treatment of Gorlin-Goltz syndrome and high-frequency BCC.
Über das Gorlin-Goltz-Syndrom
Das Gorlin-Goltz-Syndrom ist eine seltene, genetisch bedingte Krankheit, die durch konstitutionelle, vererbbare Mutationen in einem Allel des Tumorsuppressorgens PATCHED1 (PTCH1) gekennzeichnet ist, das als primärer Inhibitor des Hedgehog-Signalwegs wirkt. Dadurch bilden sich meist im Gesicht mehrere Basalzellkarzinome.
Because there are no FDA-approved drugs for Gorlin-Goltz syndrome, surgery is the standard of care. It may happen that people with severe Gorlin-Goltz syndrome have to undergo up to 30 surgeries per year, which often have to be repeated and may be associated with scarring and disfigurement. It is estimated that around 10,000 people in the United States and one in 31,000 people are affected by Gorlin-Goltz syndrome. Gorlin-Goltz Syndrome is also referred to as Gorlin Syndrome, Basal Cell Nevus Syndrome (BCNS), and Naso-Bold Cell Carcinoma Syndrome (NBCCS).
About radiofrequency basal cell carcinoma (BCC)
High-frequency BCC, like Gorlin-Goltz syndrome, is a rare disease characterized by the development of an unusually high BCC number. In contrast to patients with Gorlin-Goltz syndrome, patients with radiofrequency BCC are not born with a germ-line mutation in the PTCH1 gene and do not suffer from the other systemic manifestations of Gorlin-Goltz syndrome. The standard of care for patients with radiofrequency BCC is surgery.